phase

Ultragenyx Announces Progress Across Broad Gene Therapy Portfolio and Positive Longer-Term Data from Multiple Phase 1/2 Gene Therapy Studies

Durable and Clinically Meaningful Responses Reported from Phase 1/2 Studies of DTX401 for GSDIa and DTX301 for OTC

Phase 3 Studies for DTX401 and DTX301 to Begin in 2021

IND for UX701 for Wilson Disease Submitted; Expect to Enter Clinic in First Half 2021 using AAV Drug Product Made by HeLa PCL Platform

NOVATO, Calif. , Jan. 08, 2021 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive longer-term safety and efficacy data from the first three cohorts of the ongoing Phase 1/2 studies of DTX401, an investigational adeno-associated virus (AAV) gene therapy for Glycogen Storage Disease Type Ia (GSDIa), and DTX301, an AAV gene therapy for ornithine transcarbamylase (OTC) deficiency. In addition, dosing is nearing completion for the prophylactic steroid cohorts in both studies. Discussions with regulatory agencies continue to

COVID-19 vaccine by Reynolds subsidiary advances to scientific trial phase | Neighborhood

Kentucky Bioprocessing jobs receiving success from the research by mid-2021.

“Kentucky Bioprocessing has spent just about 15 a long time creating its plant-centered know-how to make scalable biopharmaceutical remedies that could foster far better entry to vaccines all-around the entire world,” Hugh Haydon, the subsidiary’s founder and president, mentioned in a assertion.

“KBP’s adaptive and quick-paced pharmaceutical generation platforms could aid us to fight world wide conditions at scale.”

A single important opportunity edge for the Kentucky Bioprocessing vaccine is that it has the potential to be stable at space temperature, compared with ultra-chilly settings for the Pfizer vaccine and freezing temperature for the possible Moderna vaccine.

There is certainly also the potential for output of the vaccine’s lively elements in about six weeks, as opposed with numerous months using common approaches.

Investigate on very similar plant-dependent know-how also has moved into a human clinical trial for a possible seasonal